Friedreich Ataxia Scientific News

Friedreich Ataxia and close related scientific news. Topics related to rare diseases.

Saturday, March 21, 2026

Friedreich ataxia transcriptomic dysregulation and identification of cell type-specific biomarkers: A systematic review and meta-analysis

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Friedreich ataxia transcriptomic dysregulation and identification of cell type-specific biomarkers: A systematic review and meta-analysis Ma...

The multifaceted nature of Friedreich ataxia: strategies for comprehensive patient care.

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Raza, M. L., Rawalia, M. A., Fatima, Z., & Zehra, H. (2026). The multifaceted nature of Friedreich ataxia: strategies for comprehensive ...
Monday, March 16, 2026

Design Therapeutics Maps H2 Data Readouts for RESTORE-FA

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MarketBeat. Sun, March 15, 2026. RESTORE-FA targets a demonstrable increase in endogenous frataxin (measured in mRNA and protein, including ...
Thursday, March 12, 2026

Biallelic Truncating DNAH14 Variant in Siblings with Neurodevelopmental Disorder and Predominant Ataxia: Clinical Report and Literature Review

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Baris S, Dogan M, Terali K, Gezdirici A, Eroz R, Yucel PP, Kilic H, Yavas C, Yildirim G, Baris I. Biallelic Truncating DNAH14 Variant in Sib...

Hereditary Ataxias: From Pathogenesis and Clinical Features to Neuroimaging, Fluid, and Digital Biomarkers-A Scoping Review

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Bernardi E, López-Lombardía Ó, Olmedo-Saura G, Pagonabarraga J, Kulisevsky J, Pérez-Pérez J. Hereditary Ataxias: From Pathogenesis and Clini...

Quantifying Placebo Effects in Hereditary Ataxia Trials: A Meta-Analysis of Scale for the Assessment and Rating of Ataxia (SARA) Score Changes

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Petit E, Beaubois-Gandoin A, Durr A, du Montcel ST, Coarelli G. Quantifying Placebo Effects in Hereditary Ataxia Trials: A Meta-Analysis of ...
Wednesday, March 11, 2026

Pathological frataxin deficiency in mice causes tissue-specific alterations in iron homeostasis

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Pazos-Gil M, Medina-Carbonero M, Sanz-Alcázar A, Portillo-Carrasquer M, Oliveira-Jorge L, Hernández G, Sánchez M, Delaspre F, Cabiscol E, Ro...

Friedreich-Ataxie: Die häufigste autosomal-rezessiv vererbte Ataxie [Friedreich's ataxia: The most common autosomal recessive inherited ataxia]

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Erdlenbruch F. Friedreich-Ataxie: Die häufigste autosomal-rezessiv vererbte Ataxie [Friedreich's ataxia: The most common autosomal reces...

Coexistence of Friedreich's Ataxia and Esophageal Cancer: A Case Report

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Mehrban A, Babamahmoodi A, Hamidian MT, Amiri B, Ramzani P, Karimi M. Coexistence of Friedreich's Ataxia and Esophageal Cancer: A Case R...

Therapeutic Potential of Deferiprone-Resveratrol Hybrid (DFP-RVT) Against Hepatic Iron Overload in β-Thalassemia Mice: A Proteomic Analysis

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Maneekesorn S, Yingchutrakul Y, Simanon N, Kumsab J, Butkinaree C, Moonmuang S, Li J, Charoenkwan P, Koonyosying P, Paradee N, Srichairatana...
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